Validation and reliability of a disease‐specific quality of life measure (the TranQol) in adults and children with thalassaemia major

Abstract

SummaryThis study aimed to demonstrate the validity, reliability and responsiveness of a new disease-specific quality of life (QoL) questionnaire for children and adults with thalassaemia major, the Transfusion-dependent QoL questionnaire (TranQol). 106 participants (51 adults and 55 children) were recruited from six North American thalassaemia treatment centres with a mean age of 20Á7 years (standard deviation [SD] 9, range 7-51 years). The mean total TranQol score was 71 (SD 17, 32-97) on a scale of 0-100. Patients with co-morbidities had significantly lower scores (63 vs. 75, P = 0Á001). TranQol scores showed substantial agreement (P < 0Á001) with the Health Utilities Index Mark 3 (all patients, r = 0Á65), the Pediatric QoL (children, r = 0Á77) and the Short Form (36) physical (adults, r = 0Á69) and mental summary scores (r = 0Á76). In the subgroup who rated their QoL as better, there was a 4Á0 point (SD 9Á0) improvement in TranQol scores, from baseline of 67Á1-71Á1 one week later (P = 0Á008). Test-retest reliability was excellent (intra-class correlation coefficient, 0Á93). The TranQol was valid, with acceptable correlation for all administered measures and was reliable and responsive to change. The TranQol can be incorporated into future studies of thalassaemia major.

Health related quality of life among children with transfusion dependent β-thalassaemia major and haemoglobin E β-thalassaemia in Sri Lanka: a case control study

“…HbE beta-thalassaemia Beta-thalassaemia major In this study we assessed the HRQoL using the Paediatric Quality of Life Inventory Version 4.0 (PedsQL 4.0) Generic core scales. We used this questionnaire instead of the recently validated disease specific quality of life questionnaire designed for transfusion dependent thalassaemia (Transfusion-dependent QoL questionnaire-TranQol) due to number of reasons [28]. Firstly, a validated Sinhalese translation is available only for PedsQL 4.0 questionnaire.…”

Development of a patient‐reported outcomes symptom measure for patients with nontransfusion‐dependent thalassemia (NTDT‐PRO©)

“…General anemia/HRQoL instruments may not be useful for patients with NTDT as they do not include the individual patient experience of symptom severity. Disease‐specific HRQoL PRO tools are currently available for patients with TDT . However, available TDT tools, such as the TranQol and STQOLI, are not appropriate for patients with NTDT; patients with TDT receive transfusions more frequently, and report a higher frequency of hospitalization, resulting in a different impact on HRQoL and perceived burden of disease.…”

“…Patient‐reported outcome (PRO) measures, including HRQoL, are important tools for determining patient health impairment and selecting appropriate treatment. Although disease‐specific PRO questionnaires have been developed for patients with TDT (TranQol; Specific Thalassemia Quality of Life Instrument, STQOLI), there are currently no disease‐specific PRO tools available to assess symptoms related to chronic anemia experienced by patients with NTDT.…”

Thalassaemia in children: from quality of care to quality of life

“…The QOL seems to be more affected in children from low-income and middle-income countries (such as Egypt and Thailand) compare to their North American counterparts 37 38. Additional areas of concern include anxiety and depression in patients.…”